Idebenone is a synthetic short-chain benzoquinone and a substrate for the enzyme NAD(P)H:quinone oxidoreductase (NQO1) capable of stimulating mitochondrial electron transport and supplementing cellular energy levels. Idebenone was initially developed by Takeda Pharmaceutical Company for the treatment of Alzheimer’s disease and other cognitive defects. This has been met with limited success. The Swiss company Santhera Pharmaceuticals has started to investigate it for the treatment of neuromuscular diseases. In 2010, early clinical trials for the treatment of Friedreich’s ataxia and Duchenne muscular dystrophy have been completed. In clinical trials, Idebenone (Raxone/Catena) had a positive impact on a measurement of respiratory function (Peak Expiratory Flow, or PEF) in non-ambulatory Duchenne muscular dystrophy patients who were not taking steroids. As of December 2013 the drug is not approved for these indications in North America or Europe. It is approved for the treatment of Leber's hereditary optic neuropathy (LHON) in Europe. Idebenone (Raxone) is indicated for the treatment of visual impairment in adolescent and adult patients with Leber’s Hereditary Optic Neuropathy (LHON). Because the number of patients with Leber's hereditary optic neuropathy is low, the disease is considered ‘rare’, and Raxone was designated an ‘orphan medicine’ on 15 February 2007. Idebenone is thought to help improve production of energy by restoring mitochondrial function, thereby preventing the cellular damage and the loss of sight seen in LHON. Idebenone is a rapidly absorbed, safe and well-tolerated drug and is currently the only clinically proven treatment option for Leber's hereditary optic neuropathy (LHON) patients.

Edoxaban (DU-176b, trade names Savaysa, Lixiana) is a selective factor Xa inhibitor reduces thrombin generation and thrombus formation and is an orally bioavailable anticoagulant drug. It was developed by Daiichi Sankyo to reduce the risk of stroke and systemic embolism in patients with nonvalvular atrial fibrillation (NVAF) and for the treatment of deep vein thrombosis and pulmonary embolism following 5-10 days of initial therapy with a parenteral anticoagulant.

Eliglustat, marketed by Genzyme as CERDELGA, is a glucosylceramide synthase inhibitor indicated for the long-term treatment of type 1 Gaucher disease who are CYP2D6 extensive metabolizers, intermediate metabolizers, or poor metabolizers (PMs) as detected by an FDA-cleared test.

Eslicarbazepine acetate is a third generation antiepileptic drug indicated for the treatment of partial-onset seizures. Structurally, it belongs to the dibenzazepine family and is closely related to carbamazepine and oxcarbazepine. Eslicarbazepine acetate was developed by scientists in Portugal. Its main mechanism of action is by blocking the voltage-gated sodium channel. Eslicarbazepine acetate is a pro-drug that is rapidly metabolized almost exclusively into eslicarbazepine (S-licarbazepine), the biologically active drug. It has a favorable pharmacokinetic and drug-drug interaction profile. However, it may induce the metabolism of oral contraceptives and should be used with caution in females of child-bearing age.

Sapropterin dihydrochloride, the active pharmaceutical ingredient in Kuvan Tablets, is a synthetic preparation of the dihydrochloride salt of naturally occurring tetrahydrobiopterin (BH4). Kuvan is indicated to reduce blood phenylalanine (Phe) levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU). Kuvan is to be used in conjunction with a Phe-restricted diet. Kuvan has received orphan drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMEA). Kuvan is a synthetic form of BH4, the cofactor for the enzyme phenylalanine hydroxylase (PAH). PAH hydroxylates Phe through an oxidative reaction to form tyrosine. In patients with PKU, PAH activity is absent or deficient. Treatment with BH4 can activate residual PAH enzyme, improve the normal oxidative metabolism of Phe, and decrease Phe levels in some patients.